This vector-based gene therapy encodes antibodies that block the stimulation of thyroid-stimulating hormone receptors (TSHR), which when activated can lead to hyperthyroidism. Graves’ disease is the most common form of hyperthyroidism, and is caused by stimulation of the TSHR by thyroid receptor stimulating antibodies (TrABs) and afflicts about 1.2 percent of Americans. Analysts project the global thyroid disorder treatment market to reach nearly $3 billion by 2022, due to an increase in thyroid disease cases along with growing awareness of thyroid disorders. Available medications for hyperthyroidism that block thyroid hormone production do not achieve lasting remission and require repeated administration. Other treatments include surgery or radioactive iodine, both of which may have risks.
Researchers at the University of Florida have developed a one-time treatment using an rAAV gene therapy vector that delivers anti-TSHR antibodies to reduce or block TSHR stimulation and prevent hyperthyroidism. This therapy can treat Graves’ disease, Grave’s eye disease (orbitopathy), or thyroid cancer where it is also desirable to block TSHR action.
Gene therapy using rAAV that produces anti-TSHR antibodies to treat diseases caused by hyperthyroidism
This gene therapy utilizes a recombinant adeno-associated virus to deliver a gene leading to the production of an anti-TSHR antibody or antibody fragment into a patient’s cells resulting in the production of anti-TSHR antibodies. This treatment results in the long-term production of the anti-TSHR antibody following a single administration. The antibodies the cell produces block TSHRs, preventing hyperthyroidism.